WO2023122532A3 - Compositions and methods for engineering treg cells for treatment of diabetes - Google Patents

Compositions and methods for engineering treg cells for treatment of diabetes Download PDF

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Publication number
WO2023122532A3
WO2023122532A3 PCT/US2022/081929 US2022081929W WO2023122532A3 WO 2023122532 A3 WO2023122532 A3 WO 2023122532A3 US 2022081929 W US2022081929 W US 2022081929W WO 2023122532 A3 WO2023122532 A3 WO 2023122532A3
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Prior art keywords
edited
engineering
treg cells
dual
locus
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PCT/US2022/081929
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French (fr)
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WO2023122532A2 (en
Inventor
Jane BUCKNER
David J. Rawlings
Peter J. Cook
Soo Jung Yang
Tom WICKHAM
Chandra Patel
Gene Uenishi
Philippe KIEFFER-KWON
Original Assignee
Seattle Children's Hospital (dba Seattle Children's Research Institute)
Benaroya Research Institute At Virginia Mason
Gentibio, Inc.
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Application filed by Seattle Children's Hospital (dba Seattle Children's Research Institute), Benaroya Research Institute At Virginia Mason, Gentibio, Inc. filed Critical Seattle Children's Hospital (dba Seattle Children's Research Institute)
Priority to IL313581A priority Critical patent/IL313581A/en
Priority to CA3242057A priority patent/CA3242057A1/en
Priority to AU2022420487A priority patent/AU2022420487A1/en
Publication of WO2023122532A2 publication Critical patent/WO2023122532A2/en
Publication of WO2023122532A3 publication Critical patent/WO2023122532A3/en

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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/461—Cellular immunotherapy characterised by the cell type used
    • A61K39/4611—T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
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    • A61K39/4621—Cellular immunotherapy characterized by the effect or the function of the cells immunosuppressive or immunotolerising
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/463—Cellular immunotherapy characterised by recombinant expression
    • A61K39/4632—T-cell receptors [TCR]; antibody T-cell receptor constructs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/46433—Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86—Viral vectors
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    • C07K2319/00—Fusion polypeptide
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    • C12N2800/00—Nucleic acids vectors
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  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
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  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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Abstract

Described herein are compositions and methods for engineering Treg cells for treatment of diabetes. The engineered Treg cells provided herein may be dual-edited (i.e., edited in two different loci in the cell genome), a first locus being the FOXP3 locus and the second locus being the TRAC locus. The engineering of dual-edited Treg cells as provided here may include selective expansion of dual-edited cells using a ligand that initiates and/or maintains IL-2 signal transduction in dual-edited cells. The engineering of dual-edited Treg cells as provided here may stably express FoxP3 and an exogenous TCR.
PCT/US2022/081929 2021-12-21 2022-12-19 Compositions and methods for engineering treg cells for treatment of diabetes WO2023122532A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
IL313581A IL313581A (en) 2021-12-21 2022-12-19 Compositions and methods for engineering treg cells for treatment of diabetes
CA3242057A CA3242057A1 (en) 2021-12-21 2022-12-19 Compositions and methods for engineering treg cells for treatment of diabetes
AU2022420487A AU2022420487A1 (en) 2021-12-21 2022-12-19 Compositions and methods for engineering treg cells for treatment of diabetes

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US202163292125P 2021-12-21 2021-12-21
US63/292,125 2021-12-21
US202263363918P 2022-04-29 2022-04-29
US63/363,918 2022-04-29
US202263364285P 2022-05-06 2022-05-06
US63/364,285 2022-05-06
US202263378928P 2022-10-10 2022-10-10
US63/378,928 2022-10-10
US202263384830P 2022-11-23 2022-11-23
US63/384,830 2022-11-23

Publications (2)

Publication Number Publication Date
WO2023122532A2 WO2023122532A2 (en) 2023-06-29
WO2023122532A3 true WO2023122532A3 (en) 2023-08-03

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PCT/US2022/081929 WO2023122532A2 (en) 2021-12-21 2022-12-19 Compositions and methods for engineering treg cells for treatment of diabetes

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AU (1) AU2022420487A1 (en)
CA (1) CA3242057A1 (en)
IL (1) IL313581A (en)
WO (1) WO2023122532A2 (en)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110151447A1 (en) * 2007-11-06 2011-06-23 Children's Medical Center Corporation Method to produce induced pluripotent stem (ips) cells from non-embryonic human cells
WO2020264039A1 (en) * 2019-06-27 2020-12-30 Seattle Children's Hospital (dba Seattle Children's Research Institute) Artificial antigen-specific immunoregulatory t (airt) cells

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110151447A1 (en) * 2007-11-06 2011-06-23 Children's Medical Center Corporation Method to produce induced pluripotent stem (ips) cells from non-embryonic human cells
WO2020264039A1 (en) * 2019-06-27 2020-12-30 Seattle Children's Hospital (dba Seattle Children's Research Institute) Artificial antigen-specific immunoregulatory t (airt) cells

Also Published As

Publication number Publication date
AU2022420487A1 (en) 2024-07-04
CA3242057A1 (en) 2023-06-29
IL313581A (en) 2024-08-01
WO2023122532A2 (en) 2023-06-29

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